Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with ASD. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to study ASD development and screen agents for assessing their effect on progression or symptoms of an ASD.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal involved in cardiovascular disease. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. The invention also provides zinc finger nucleases that target chromosomal sequences involved in cardiovascular disease and the nucleic acids encoding said zinc finger nucleases. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with cognitive disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with a secretase disorder. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with Parkinson's disease. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to study PD development and screen agents for assessing their effect on progression or symptoms of PD.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with neurodevelopmental disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences involved in tumor suppression. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. The invention also provides zinc finger nucleases that target chromosomal sequence involved in tumor suppression and the nucleic acids encoding the zinc finger nucleases. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences involved in tumor suppression.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with cognitive disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences associated with cognitive disorders.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated ALS. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding inflammation-related proteins. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences encoding inflammation-related proteins.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with trinucleotide repeat expansion disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with neurotransmission disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: Foot and mouth disease (FMD) viruses which are able to grow on BHK-21 cells in suspension are described herein. The new viruses are recombinant chimeric viruses formed by replacing the outer capsid coding region of a first FMDV strain, which has previously been shown to be an effective vaccine strain, with the outer capsid coding region of a second FMDV strain. The outer capsid coding region of the second FMDV strain is also modified to introduce a heparan sulphate proteoglycan (HSPG) binding site. The chimeric viruses are then used as seed viruses in the production of inactivated vaccine antigens which have been tailored for specific outbreak situations or locality. The invention also relates to the product of expression of the chimeric FMD viruses and to uses therefor, such as to form antigenic, immunological or vaccine compositions for prevention of FMD.

Abstract: The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with nociception or taste disorders. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.

Abstract: Cultures of cells immunoreactive for glial fibrillary acidic protein (GFAP), as well as for the intermediate filament marker nestin were grown in a medium including epidermal growth factor (EGF) and serum. The cultured cells had the morphology of astroglial cells. The cells can be proliferated in adherent or suspension cultures. Depending on the culture conditions, the cells can be induced to differentiate to neurons or glial cells. The cultures can be expanded over a large number of passages during several months, and survive, express an astroglial phenotype and integrate well after transplantation into both neonatal and adult rat forebrain.

Abstract: The disclosure provides a novel anti-angiogenesis fusion protein. The present invention combines a chimeric vascular endothelial cell growth factor (VEGF) receptor or a fragment thereof with a multimerizing component, which have a superior binding capacity with human VEGF and placental growth factor (PIGF). The fusion protein has improved stability, prolonged half-life and the ability to form multivalent interactions with VEGF, and can be used for anti-angiogenesis, treating VEGF related diseases and inhibiting tumor growth.

Abstract: It is an object of the present invention to provide a non-human gene-disrupted animal with a disrupted ADAM11 gene. According to the present invention, a non-human gene-disrupted animal, wherein either one of or both alleles of an ADAM11 gene are disrupted, is provided.

Abstract: Methods of forming ex vivo cell cultures comprising differentiated mesenchymal lineage cells are disclosed. These methods comprise a) providing a cell culture comprising a plurality of mesenchymal stem cells (MSCs); b) subjecting the MSCs to hypoxic conditions; and c) subsequent to b), subjecting the MSCs to normoxic conditions. Enhanced differentiation of various mesenchymal lineage cells can be achieved for mammalian cells such as murine cells or human cells.

Abstract: The present invention relates to GPR1 25 as a marker of stem and progenitor cells, including multipotent adult spermatogonial-derived stem cells (MASCs), spermatogonial stem and progenitor cells, skin stem or progenitor cells, intestinal stem or progenitor cells, neural stem or progenitor cells, and cancer stem cells. The invention provides, inter alia, methods for enriching or isolating GPR125-positive stem or progenitor cells, methods for detecting GPR125-positive stem or progenitor cells, methods for culturing GPR125-positive stem or progenitor cells, purified GPR125-positive stem or progenitor cells, therapeutic compositions containing purified GPR125-positive stem or progenitor cells, methods for targeting therapeutic agents to GPR125-positive stem and progenitor cells, and methods of treatment comprising administering GPR125-positive stem and progenitor cells, or differentiated cells derived therefrom, to subjects in need thereof.

Abstract: Methods and cell lines for overexpressing functional gamma-carboxylated proteins are disclosed by way of genetically engineered cell lines which over-express VKORC1. Also disclosed is the antisense inhibition of expression of calumenin in conjunction with overexpression of VKORC1 which also increases expression of functional gamma-carboxylated proteins. Gamma-carboxylated proteins of interest may include blood coagulation factors such as human clotting factors IX and VII.

Abstract: The present invention is directed to the identification of a biomarker specifically located in the plasma membrane of pancreatic beta cells. It was selected by a Systems Biology approach on Massively Parallel Signal Sequencing datasets obtained in human islets and Affymetrix microarray datasets on human islets, purified rat primary beta and non beta cells and insulinoma cells. Based on a set of specific features the biomarker is a unique candidate for imaging and targeting strategies to study the pancreatic beta cell mass in health and disease (T1 D, T2D, pancreatic cancers, obesity, islet transplantation, beta cell regeneration).

Abstract: The present invention relates to polynucleotides encoding immunogenic HIV type C polypeptides. Uses of the polynucleotides in applications including DNA immunization, generation of packaging cell lines, and production of HIV Type C proteins are also described.

Abstract: The present invention relates to the cloning, identification and characterization of the unique and entire genomic sequences encoding new porcine DC-SIGN and LSECtin proteins, including the novel nucleotide sequences of the full-length cDNA and genes of both pDC-SIGN gene and pLSECtin. Also provided are the nucleic acid molecules encoding newly discovered porcine ICAM-3 isoforms from porcine monocyte-derived dendritic cells and the use thereof. Specifically, the invention is drawn to an isolated nucleic acid molecule comprising a nucleotide sequence encoding one or more of porcine DC-SIGN, porcine ICAM-3, porcine LSECtin, a complement of the nucleotide sequence or a functional, defined portion of the nucleotide sequence or a protein fusion product linked to a protein that may be of porcine or human origin.

Abstract: The invention provides compositions (e.g., pharmaceuticals, formulations) and methods for ameliorating (e.g., preventing or treating) an anemia and/or stimulating erythropoiesis and/or EPO erythropoietin synthesis. The invention provides compositions comprising a chimeric protein artificial transcription factor comprising a plurality of (multiple) protein DNA-binding domains, e.g., zinc finger binding domains, specific for the promoter region of an erythropoietin (EPO) gene; a consensus nuclear localization protein sequence; a cell-penetrating peptide sequence; and a transcription activation domain.

Abstract: Disclosed herein are methods and compositions for inactivating a glutamine synthetase (GS) gene, using fusion proteins comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding said fusion proteins are also provided, as are cells comprising said polynucleotides and fusion proteins.

Abstract: Disclosed are compositions and methods for increasing the longevity of a cell culture and permitting the increased production of proteins, preferably recombinant proteins, such as antibodies, peptides, enzymes, growth factors, interleukins, interferons, hormones, and vaccines. Cells transfected with an apoptosis-inhibiting gene or vector, such as a triple mutant Bcl-2 gene, can survive longer in culture, resulting in extension of the state and yield of protein biosynthesis. Such transfected cells exhibit maximal cell densities that equal or exceed the maximal density achieved by the parent cell lines. Transfected cells can also be pre-adapted for growth in serum-free medium, greatly decreasing the time required to obtain protein production in serum-free medium. In certain methods, the pre-adapted cells can be used for protein production following transformation under serum-free conditions. The method preferably involves eukaryotic cells, more preferably mammalian cells.

Abstract: The present invention provides for the identification of autoreactive T cell populations from individuals having autoimmune diseases, such as multiple sclerosis and EAE. Peptoids recognized by autoreactive T cells can be used to identify various types of autoimmune disease, and can also be used to target therapies against such populations.

Abstract: The present invention provides compositions and methods for the production of glycoproteins with enhanced sialylation. In particular, the invention provides cell lines comprising disrupted sialidase expression and methods of using the cell lines to produce glycoproteins with enhanced sialylation.

Abstract: A new method for selecting clones and recloning mammalian cells which are of importance for the production of biopharmaceuticals, preferably hamster or mouse myeloma cells, with a high degree of automation and throughput. The invention relates to methods of depositing and replicating single cell clones of the cells in question. The invention also relates to methods of preparing proteins using cells which have been obtained and replicated by single cell deposition as well as compositions which allow the replication of single cells.

Abstract: The present invention relates to a method for producing a preantral follicle-derived embryonic stem cell and a preantral follicle-derived embryonic stem cell. The present method comprises the steps of (a) obtaining a preantral follicle from mammalian ovaries; (b) growing the preantral follicle in vitro; (c) maturing an oocyte in vitro present in the cultured preantral follicle; (d) activating the matured oocyte for parthenogenesis; (e) culturing the activated oocyte to form a blastocyst; and (f) culturing inner cell mass (ICM) cells of the blastocyst to produce the preantral follicle-derived embryonic stem cell.

Abstract: The present invention provides compounds and methods for the treatment and prophylaxis of renal disease and inflammation. In particular the invention provides methods for the treatment of kidney disease and failure through the administration of compounds which function as inhibitors of TLR2 function and expression.

Abstract: The present invention concerns the production, specific expression, and permanent release of canine interleukines, especially interleukin-2 (cIL-2) or interleukin-12 (cIL-12), from transfected BHK Tet-On cells, as well as the utilization of lymphokin-activated killer cells which were generated therewith for the tumor therapy in dogs.

Abstract: The invention is directed to a method for altering the ion conductivity of a membrane. The method comprises inserting a biological photoreceptor into the membrane. The biological photoreceptor is configured to act as a light-controlled ion channel. The photoreceptor used comprises an apoprotein and a light-sensitive polyene covalently bound to the apoprotein, wherein the polyene interacts with the apoprotein and functions as a light-sensitive gate.

Abstract: Disclosed is a method for preparing an embryonic stem cell (ESC)-like cell, which includes the steps of: (a) obtaining a first cell population from a mammalian tissue or body fluid, wherein the first cell population comprises adult stem cells; (b) obtaining a second somatic cell population from a mammalian tissue, wherein the mammalian tissue is different from the mammalian tissue in step (a) and the second cell population is different from the first cell population; (c) coculturing the first cell population and the second cell population in a medium for a period of time sufficient to form a colony from either the first cell population or the second cell population; and (d) subculturing a cell from the colony in a medium for a period time sufficient to prepare the ESC-like cell.

Abstract: A method and oligonucleotides for targeted nucleotide exchange of a duplex DNA sequence, wherein the donor oligonucleotide contains at least two modified nucleotides, at least one of which is a propynylated purine and/or pyrimidine and at least one of which is a LNA having a higher binding affinity compared to naturally occurring A, C, T or G and/or binds stronger to a nucleotide in an opposite position in the first DNA sequence as compared to a naturally occurring nucleotide complementary to the nucleotide in the opposite position in the first DNA sequence.

Abstract: An isolated polynucleotide molecule includes a DNA sequence encoding an infectious RNA molecule encoding a modified live viral strain of an Equine arteritis virus, wherein the DNA sequence is SEQ ID NO:1 or a degenerate variant thereof. Also provided are transformed or transfected host cells including that sequence, vectors including the sequence, and isolated infectious RNA molecules encoded by the sequence. Further, a modified DNA sequence encoding an infectious RNA molecule encoding a modified live viral strain of an Equine arteritis virus is provided wherein the DNA sequence is SEQ ID NO:2 or a degenerate variant thereof, including a si lent point mutation allowing distinguishing the modified sequence from the parent and other strains of Equine arteritis virus.

Abstract: Antibody expression vectors and plasmids can incorporate various antibody gene portions for transcription of the antibody DNA and expression of the antibody in an appropriate host cell. The expression vectors and plasmids have restriction enzyme sites that facilitate ligation of antibody-encoding DNA into the vectors. The vectors incorporate enhancer and promoter sequences that can be varied to interact with transcription factors in the host cell and thereby control transcription of the antibody-encoding DNA. A kit can incorporate these vectors and plasmids.

Abstract: A method and oligonucleotides for targeted nucleotide exchange of a duplex DNA sequence, wherein the donor oligonucleotide contains at least one modified nucleotide which is a LNA having a higher binding affinity compared to naturally occurring A, C, T or G and/or binds stronger to a nucleotide in an opposite position in the first DNA sequence as compared to a naturally occurring nucleotide complementary to the nucleotide in the opposite position in the first DNA sequence.

Abstract: The current invention comprises a nucleic acid encoding the amino acid sequence of the C-terminal part of the CH3-domain of an immunoglobulin of the class IgA or IgG, or the C-terminal part of the CH4-domain of an immunoglobulin of the class IgE or IgM, wherein the glycine-lysine-dipeptide comprised in the amino acid sequence of the C-terminal part of the CH3- or CH4-domain is encoded by the nucleic acid ggaaaa, or the nucleic acid ggcaaa, or the nucleic acid gggaaa, or the nucleic acid gggaag, or the nucleic acid ggcaag, or the nucleic acid ggaaag.

Abstract: The present invention relates to preventing or delaying programmed cell death by expressing one or more anti-apoptotic polypeptides in a cell expressing recombinant Factor VIII such that programmed cell death in the cell is prevented or delayed. The present invention also relates to increasing production of recombinant Factor VIII by expressing one or more anti-apoptotic polypeptides in a cell such that production of recombinant Factor VIII by the cell is increased. Recombinant cells useful for producing Factor VIII.

Abstract: Modified factor VII polypeptides and uses thereof are provided. Such modified FVII polypeptides include Factor VIIa and other forms of Factor VII. Among modified FVII polypeptides provided are those that have altered activities, typically altered procoagulant activity, including increased procoagulant activities. Hence, such modified polypeptides are therapeutics.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

Abstract: The present invention generally relates to humanized VEGF and non-human transgenic animals expressing it. The transgenic animals are also useful to study VEGF-related therapies.

Abstract: The present invention relates to a cell for the production of an antibody molecule such as an antibody useful for various diseases having high antibody-dependent cell-modulated cytotoxic activity, a fragment of the antibody and a fusion protein having the Fc region of the antibody or the like, a method for producing an antibody composition using the cell, the antibody composition and use thereof.

Abstract: Particular aspects provide novel recombinant cells and cell lines (e.g., macrophage cell lines) that are permissive for propagation of porcine reproductive and respiratory syndrome virus (PRRSV) propagation in vitro or in vivo. In certain aspects, novel nucleic acid sequences encoding porcine sialoadhesin were transfected into existing macrophage cell-lines from other species, rendering them permissive to PRRSV infection, and suitable for propagation of PRRSV. Particular aspects provide exemplary recombinant cloned cell lines that support the replication of PRRSV, with an obtainable PRRSV titre of between 2×105/ml and 2×106/ml. Additional aspects provide novel nucleic acid sequences and polymorphisms thereof that encode for porcine sialoadhesin. Further aspects provide PRRSV propagation and preparation methods using the novel recombinant cell lines, and methods for PRRSV antigen and vaccine production using same.

Abstract: The present invention provides core promoter motif ten elements (MTE) and core promoter constructs comprising the MTEs and an initiator element (Inr) in combination with one or both of a TATA box and a downstream promoter element (DPE) which increases gene expression over the strongest known core promoters. Particularly, an optimized or super core promoter is provided which comprises Inr, MTE, TATA box and DPE elements. The present invention also provides expression vectors and host cells comprising the core promoter constructs. Additionally, methods of increasing production of a protein using the core promoter constructs are provided.

Abstract: The present invention provides vectors that contain and express in vivo the genes encoding VP2 and VP5 of African Horse Sickness Virus or an epitope thereof that elicits an immune response in a horse against African horse sickness virus, compositions comprising said vectors, methods of vaccination against African horse sickness virus, and kits for use with such methods and compositions.

Abstract: The present invention provides a process of isolation, proliferation and/or maintenance of mesenchymal stem cells (MSCs). The invention further provides a culture medium for proliferation and/or maintenance of human mesenchymal stem cells in xeno-free conditions. The culture medium provided in the present invention proliferates and/or maintains mesenchymal stem cell expansion while maintaining a multipotent phenotype.

Abstract: Monoclonal antibodies which specifically bind human CD23, the low affinity receptor for IgE (FceRII/CD23), and contain either a human gamma-1 or human gamma-3 constant domain, are disclosed. The antibodies are useful for modulating or inhibiting induced IgE expression. Accordingly, they have practical utility in the treatment or prophylaxis of disease conditions wherein inhibition of induced IgE production is therapeutically desirable, including allergic conditions, autoimmune diseases and inflammatory diseases.

Abstract: Methods of preparing a NELL peptide are disclosed.